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CORPORATE PRESENTATION 4 JUNE 2019

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Page 1: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

CORPORATE PRESENTATION4 JUNE 2019

Page 2: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Important notice

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You must read the following before continuing. The following applies to this document and the information provided in this presentation by Medivir AB (publ) (the “Company”) or any person onbehalf of the Company and any other material distributed or statements made in connection with such presentation (the “Information”), and you are therefore advised to carefully read thestatements below before reading, accessing or making any other use of the Information. In accessing the Information, you agree to be bound by the following terms and conditions.

The Information does not constitute or form part of, and should not be construed as, an offer of invitation to subscribe for, underwrite or otherwise acquire, any securities of the Company or asuccessor entity or any existing or future subsidiary or affiliate of the Company, nor should it or any part of it form the basis of, or be relied on in connection with, any contract to purchase orsubscribe for any securities of the Company or any of such subsidiaries or affiliates nor shall it or any part of it form the basis of or be relied on in connection with any contract or commitmentwhatsoever. Specifically, this presentation does not constitute a “prospectus” within the meaning of the U.S. Securities Act of 1933, as amended.

The Information may not be reproduced, redistributed, published or passed on to any other person, directly or in directly, in whole or in part, for any purpose. The Information is not directed to, orintended for distribution to or use by, any person or entity that is a citizen or resident of, or located in, any locality, state, country or other jurisdiction where such distribution or use would be contraryto law or regulation or which would require any registration or licensing within such jurisdiction. The Information is not for publication, release or distribution in the United States, Australia, Canada orJapan, or any other jurisdiction in which the distribution or release would be unlawful.

All of the Information herein has been prepared by the Company solely for use in this presentation. The Information contained in this presentation has not been independently verified. Norepresentation, warranty or undertaking, express or implied, is made as to, and no reliance should be placed on, the fairness, accuracy, completeness or correctness of the Information or theopinions contained herein. The Information contained in this presentation should be considered in the context of the circumstances prevailing at that time and will not be updated to reflect materialdevelopments which may occur after the date of the presentation. The Company may alter, modify or otherwise change in any manner the content of this presentation, without obligation to notifyany person of such revision or changes.

This presentation may contain certain forward-looking statements and forecasts which relate to events and depend on circumstances that will occur in the future and which, by their nature, willhave an impact on the Company’s operations, financial position and earnings. The terms “anticipates”, “assumes”, “believes”, “can”, “could”, “estimates”, “expects”, “forecasts”, “intends”, “may”,“might”, “plans”, “should”, “projects”, “will”, “would” or, in each case, their negative, or other variations or comparable terminology are used to identify forward-looking statements. There are a numberof factors that could cause actual results and developments to differ materially from those expressed or implied in a forward-looking statement or affect the extent to which a particular projection isrealized. Factors that could cause these differences include, but are not limited to, implementation of the Company’s strategy and its ability to further grow, risks associated with the developmentand/or approval of the Company’s products candidates, ongoing clinical trials and expected trial results, the ability to commercialize existing and any future products, technology changes and newproducts in the Company’s potential market and industry, the ability to develop new products, the impact of competition, changes in general economy and industry conditions and legislative,regulatory and political factors. While the Company always intends to express its best judgment when making statements about what it believes will occur in the future, and although the Companybases these statements on assumptions that it believe to be reasonable when made, these forward-looking statements are not a guarantee of its performance, and you should not place unduereliance on such statements. Forward-looking statements are subject to many risks, uncertainties and other variable circumstances. Many of these risks are outside of the Company’s control andcould cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this presentation are made only as of the date hereof. The Companydoes not undertake, and specifically decline, any obligation to update any such statements or to publicly announce the results of any revisions to any of such statements to reflect future events ordevelopments.

Page 3: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Medivir – Recent events

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• Clinical Development focus on oncology • Birinapant/Keytruda® : completion phase I study – Q4 2018

• Birinapant/Keytruda® : start of phase II study – Q4 2018

• MIV-818: Start of phase I study – Q4 2018

• MIV-828: CD nomination – Q4 2018

• Fixed cost base reduced with two thirds

• Strong management and cost-effective virtual organization

Page 4: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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PROJECT & MECHANISM DISEASE AREA RESEARCH PRECLINICAL PHASE I PHASE II PHASE III EXCLUSIVITY

RemetinostatHDAC INHIBITOR (TOPICAL)

Cutaneous T-cell lymphoma (MF)

Basal cell carcinoma1)

BirinapantSMAC MIMETIC (INTRAVENOUS)

Solid tumors(combo with Keytruda®)

MIV-818NUCLEOTIDE DNA POLYMERASEINHIBITOR (ORAL)

Hepatocellular carcinoma

MIV-828NUCLEOTIDE BASED DNA POLYMERASEINHIBITOR (INTRAVENOUS)

Blood cancer (acute myeloid leukemia)

MIV-711CATHEPSIN K INHIBITOR (ORAL)

Osteoarthritis

Broad and robust pipeline

1) Investigator sponsored study at Stanford U.

IP: 2034

IP: 2034

Completed Ongoing

IP: Est 2039

IP: 2034

IP: 2035

IP: 2034

Page 5: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Remetinostat for early-stage MF cutaneous T-cell lymphoma

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Page 6: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MF-CTCL: orphan blood cancer indication

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• CTCL is a rare form of non-Hodgkin lymphoma primarily present in the skin. Mycosis fungoides (MF) is the most common form of CTCL

• Annual new cases; US 2,000; EU 3,000; Sweden 25

• Five-year survival: 85%; more than 16,000 US patients live with MF-CTCL

• Skin lesions and severe itching are common and affect patients quality of life

• Early stage disease lasts for long periods and requires well tolerated therapy

• Available treatments, including systemic HDAC inhibitors, have severe side effects

Cutaneous T-cell lymphoma (CTCL) affects lymphocytes (cells belonging to the immune defense system) located in the skin and typically has a chronic course.

Page 7: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Remetinostat: for treatment of early stage MF-CTCL

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• Remetinostat is a histone deacetylase (HDAC) inhibitor

• Remetinostat’s unique chemistry and topical formulation provides for activity in skin and rapid degradation in blood

• Approved HDAC inhibitors not used in early-stage MF-CTCL patients

• US orphan drug designation 600 30 90

Page 8: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Dose 1% 1x/day n=20

0.5% 2x/day n=20

1% 2x/day n=20

Lesion responses1 20% 25% 40%

Patients with clinically significantpruritus

(40%)n=8/20

(30%)n=6/20

(50%)n=10/20

Pruritus responses 38% 50% 80%

Remetinostat: clinical proof-of-concept phase II MF-CTCL study

Well tolerated:

• No HDAC inhibitor-associated systemic adverse events

• Median time on treatment: 336 days (1% 2x/day dose)

Twelve months phase II data shows reduction in both lesions and severe itch

1) Confirmed responses based on CAILS, the Composite Assessment of Index Lesion Severity

Page 9: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Remetinostat: next steps

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• Medivir will further define a planned phase III design based on the requirements clarified by the FDA

• One phase III study expected to be sufficient for FDA

• Phase III study will enroll treatment-experienced patients

• Medivir aims to identify a business partner for the further development of remetinostat

Page 10: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Remetinostatopportunity in other skin cancers

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Page 11: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Investigator-initiated phase II study in BCC ongoing

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Study start-Tumor staging-Biopsy consent

Topical application of remetinostat under occlusion (1.0% gel t.i.d.)12 weeks f.u.

Week 4-Tumor assessment-Safety assessment

Week 8-Tumor assessment-Safety assessment-Biopsy consent

Drug product,

delivered from

Medivir

Study design

• 8-week open-label phase II clinical study to assess efficacy of remetinostat in patients with BCC

• Primary objective: Determination of suppression of BCC growth & ORR (overall response rate) using RECIST v1.1 criteria

Basal cell carcinoma

• The most common form of cancer in humans occurring in the skin• Surgery is standard of care, but there is a need for efficacious and safe treatments when surgery is impractical,

e.g. multiple lesions and/or difficult treatment sites

Investigator: Kavita Sarin, Stanford University School of Medicine in California, USA

Page 12: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Interim results presented at SID Annual meeting May 2019

• The ORR, at least a 30% decrease in longest diameter, was 64% (9/14).

• The average decrease in tumor area is 70% (n=14), while the average decrease in

longest diameter is 62% (n=14). 43% (6/14) of tumors were fully cleared.

• No systemic toxicities have been observed.

• Grade 2 eczematous local site reactions occurred in 71% (10/14) tumors treated with

topical remetinostat under bandage occlusion.

More information can be found at www.medivir.com

Page 13: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Birinapant: Uniquely potent against selected solid tumors

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Page 14: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Solid tumors: large unmet medical needs

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Colorectal cancer indication (CRC)

• The second most common cancer in women and the third in men

• Estimated new cases 2018: US: 140,000; EU: 490,000; Sweden: 6,200

• Five-year survival when metastatic: 14%

Other cancer indications• Ovarian cancer, the leading cause of mortality due a gynecologic tumor

• Estimated new cases 2018: US: 22,000; EU: 23,000 Sweden: 700

• Five-year survival: 47%

• Cervical cancer, the third most common cancer in women world-wide

• Estimated new cases 2018: US: 13,000; EU: 60,000; Sweden: 450

• Five-year survival: 62.5%

Many patients with solid tumors have few or no options and are in need of effective medicines to extend life. The immuno-oncology medicine Keytruda® on its own is not sufficiently effective in treatment of certain solid tumors.

Page 15: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Birinapant may benefit patients with inadequate response to immuno-oncology therapies

• Birinapant, a SMAC mimetic, enables tumor cell death and augments the immune system

• Great potential to improve treatment of cancers when combined with immuno-therapy

• Ongoing collaboration with Merck for a phase I/II study in solid tumors

o Joint development committee oversees the study

o Keytruda® provided at no cost by Merck

o Medivir retains full global rights to birinapant and data

Page 16: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Birinapant/Keytruda® combination - phase I/II study ongoing

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Phase II

• Dose escalation completed; December 2018: n=19

o One CRC patient has achieved partial response, which had been maintained for over 1 year

o Two patients had stable disease for 18 weeks

o Safety and tolerability: No concerns

o Phase II dose selected at 22 mg/m2

RP2D

Phase II expansion, n≤28 pts Phase I (n= 19 pts)Interpatient dose escalation ( 3+3 design)

Phase I

Phase 2 Ovarian (n=17+10)Phase I: Sequential group dose-escalation to determine safety and tolerability and establish the recommended phase II dose.

Phase II: Safety, tolerability and efficacy of the recommended dose of birinapant.

• In late December 2018 the first patient was dosed in the phase II part of the study

Page 17: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MIV-818: Nucleotide prodrug for the treatment of liver cancer

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Page 18: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Liver cancer focus: hepatocellular carcinoma (HCC) and intrahepatic cholangiocarcinoma

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• HCC is the third leading cause of cancer-related deaths worldwide

o Estimated new cases 2018: Asia: 610,000; US: 42,000; EU: 82,000; Sweden: 550

o Orphan disease in Western markets, but one of fastest growing and most deadly cancers in US

o High incidence in Asia including China - Hepatitis B & C very common

o Five-year survival: 18%

o Genetically heterogeneous leading to limited effect of molecularly targeted therapies

• Intrahepatic cholangiocarcinoma is the second most common primary liver tumor

o Median survival is only twelve months

• Existing treatment options provide very little survival benefit

Page 19: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MIV-818: prodrug for enhanced efficacy and safety in liver cancer therapy

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MIV-818

• Enhanced activity

• Selectivity for cancer

• Improved delivery to the liver

• Oral administration

• Limited systemic side effect

Troxacitabine

• Clinically active but failed due to systemic dose-limiting toxicities

Medivir prodrug

technology

Phase Ia (n=6)Intrapatient dose escalation

Phase Ib (n=24)Interpatient dose escalation

Safety and tolerability. Decision to move into phase Ib

Safety and tolerability. Decision about recommended dose for phase II

RP2DPhase I

Page 20: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MIV-828: For acutemyeloid leukemia

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Page 21: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MIV-828: Summary

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Opportunity in hematological cancers

• Better tolerated and more effective agent in patients with AML and other hematological cancers

• Initial development in relapsed/refractory AML patients

Profile of MIV-828

• Nucleotide prodrug based on one of Medivir’s proprietary areas of expertise

• Active metabolite shown to have potent anti-leukemic activity in preclinical in vivo models

• Increased activity and reduced susceptibility to pharmacologic resistance mechanisms of prodrugs

• Preclinical activity also demonstrated against T-cell lymphoma

• Expected new cases 2018: US: 19,500; EU: 43,000; Sweden: 350

• Affects all ages, median age at diagnosis 68 years in the US

• Five-year survival: 50% in younger patients (<60y) and less than 10% in patients > 70y

Acute myeloid leukemia (AML) leads to an overproduction of abnormal immature cells in the bone marrow and prevents development of normal blood cells, which could lead to anemia, bleeding episodes and a high susceptibility for severe infections.

Page 22: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MIV-711: Cathepsin K inhibitor with FDA fast track status

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Page 23: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Osteoarthritis (OA): the most common form of joint disease

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• Affects ~240m adults worldwide

• Prevalence increasing due to aging population and obesity epidemic

• Current treatments focus only on pain relief

• Large unmet medical need for a disease-modifying drug (DMOAD) with potential to slow, halt or reverse the progression of OA

• New US FDA guidelines in OA may enable pathway for accelerated regulatory approval

• New draft FDA Guidance published August 2018, focused on structural endpoints in OA development

Yuan et al. Osteoarthritis and Cartilage (2014), 22, 1077

OA kneeHealthy knee

Cathepsin K protease is involved in the breakdown of collagen I in both bone and collagen II in cartilage

Page 24: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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MIV-711: positive effects on joint structure and signalsof benefit on clinical symptoms

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Placebon=80

MIV-711 100 mg QDn=80

MIV-711 200 mg QDn=80

Femur bone area (mm2) 23.2 8.1 8.2

Cartilage thickness (mm) -0.066 0.008 -0.017

• Study design (MIV-711-201): 3 arms, 26 weeks treatment• Study design (MIV-711-202): 200 mg MIV-711, 26 additional weeks

MIV-711-201: Change from baseline vs week 26

• A trend consistently favoring MIV-711 arms in all predefined analyses of clinical outcomes, e.g. knee pain and knee function

• Safety and tolerability profile supporting advancement of MIV-711 as a disease-modifying OA drug candidate

Page 25: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Summary

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Page 26: Redeye life science day Stockholm, November 21, 2018...Other cancer indications • Ovarian cancer, the leading cause of mortality due a gynecologic tumor • Estimated new cases 2018:

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Near term value inflection points

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• MIV-818: completion phase Ia study – Q2 2019

• Birinapant/Keytruda® : futility analysis completed – Q4 2019

Strategic focus on cancer indications with high unmet need